Latest results from the COMPOSE® Phase 1/2 trial for the treatment of classical homocystinuria (HCU) using pegtibatinase, a novel investigational enzyme replacement therapy

Janet Thomas,Jaya Ganesh, Wendy Smith,Melissa Lah, David Kudrow, Jalé Güner, Sharon McDermott,Sagar A. Vaidya, Liz Wilkening, Harvey Levy,Can Ficicioglu

Molecular Genetics and Metabolism(2024)

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